Intranasal ERT in LSDs may bypass BBB

According to the study by Wolf et al. intranasal administration of Î±-l-iduronidase (IDUA) enzyme may be able to bypass the blood-brain barrier. This appears to be the case in IDUA-deficient mice after a single intranasal treatment with concentrated AldurazymeÂ®.Â Promising results in mice were also noted after intranasal treatment with an adeno-associated virus (AAV) vector expressing human IDUA. These experiments raise the possibility of non-invasive treatments for lysosomal storage disorders.